for people ages 3-11 (full criteria)
at Oakland, California and other locations
study started
completion around



This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

Official Title

Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2


Achondroplasia, Skeletal dysplasia, Endochondral ossification, ACH, Shortened proximal limbs, Fibroblast growth factor receptor 3, FGFR3, Endochondral bone formation, Short-limb disproportionate dwarfism, dwarfism, Bone disease, Bone diseases, developmental, Musculoskeletal diseases, Osteochondrodysplasia, Genetic diseases, inborn, Inborn, Infigratinib, Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg, Infigratinib 0.128 mg/kg, Infigratinib 0.25 mg/kg


You can join if…

Open to people ages 3-11

  1. Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable).
  2. Diagnosis of ACH, documented clinically and confirmed by genetic testing.
  3. At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.
  4. Ambulatory and able to stand without assistance
  5. Able to swallow oral medication.

You CAN'T join if...

  1. Hypochondroplasia or short stature condition other than ACH.
  2. In females, having had their menarche.
  3. Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH.
  4. Significant concurrent disease or condition that, in the view of the Investigator and/or Sponsor, would confound assessment of efficacy or safety of infigratinib.
  5. Current evidence of corneal or retinal disorder/keratopathy.
  6. History of malignancy.
  7. Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 and medications which increase serum phosphorus and/or calcium concentration.
  8. Treatment with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (>3 months) at any time.
  9. Treatment with a C-type natriuretic peptide (CNP) analog, fibroblast growth factor (FGF) ligand trap, or treatment targeting FGFR inhibition at any time.
  10. Regular long-term treatment (>3 weeks) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).
  11. Treatment with any other investigational product or investigational medical device for the treatment of ACH or short stature.
  12. Previous limb-lengthening surgery or guided growth surgery.
  13. Fracture within 12 months of screening.


  • UCSF Benioff Children's Hospital
    Oakland California 94618 United States
  • Stollery Children's Hospital
    Edmonton Alberta T6G 2H7 Canada


in progress, not accepting new patients
Start Date
Completion Date
QED Therapeutics, Inc.
Phase 2 research study
Study Type
About 84 people participating
Last Updated